Hus­tling to cap­ture top front­line NSCLC sta­tus, Roche scores a ‘break­through’ on Ale­cen­sa

San­dra Horn­ing, Roche CMO

The FDA has hand­ed Roche brag­ging rights to its sec­ond break­through drug des­ig­na­tion for Ale­cen­sa (alec­tinib), which is like­ly to help make the drug’s case as the new stan­dard for front-line treat­ment of ALK-pos­i­tive non-small cell lung can­cer.

BTDs are not in the least bit un­usu­al for big play­ers like Roche, which has now racked up a long string of them. But this one hits home, of­fer­ing ‘break­through’ sta­tus for this drug for pre­vi­ous­ly un­treat­ed pa­tients. Roche is fac­ing some ma­jor league com­pe­ti­tion on that score, and it won’t mind the added ben­e­fits a BTD of­fers in win­ning over reg­u­la­tors as well as in­flu­en­tial prac­ti­tion­ers.

San­dra Horn­ing ham­mered the front­line point in a state­ment. She said:

“The J-ALEX study that sup­ports the sec­ond Break­through Des­ig­na­tion for Ale­cen­sa showed su­pe­ri­or ef­fi­ca­cy ver­sus the stan­dard of care, crizo­tinib (Xalko­ri), in Japan­ese peo­ple with ad­vanced ALK-pos­i­tive dis­ease. The de­ci­sion by the FDA to grant a sec­ond break­through ther­a­py des­ig­na­tion is recog­ni­tion of the clin­i­cal­ly mean­ing­ful im­prove­ment in ef­fi­ca­cy and safe­ty that Ale­cen­sa brings to the care of peo­ple with ad­vanced ALK-pos­i­tive lung can­cer who have not re­ceived pri­or treat­ment with an ALK in­hibitor.”

A cou­ple of weeks ago No­var­tis out­lined plans to hus­tle to reg­u­la­tors with new late-stage da­ta on their can­cer drug Zyka­dia (cer­tinib), look­ing for an ap­proval to move up to first-line treat­ment of ALK-pos­i­tive non-small cell lung can­cer. In­ves­ti­ga­tors say the drug out­per­formed stan­dard chemo in pre­vi­ous­ly un­treat­ed pa­tients for pro­gres­sion-free sur­vival. There were al­so “clin­i­cal­ly mean­ing­ful” im­prove­ments in the over­all re­sponse rate and du­ra­tion of re­sponse, though that will need to be clar­i­fied when the da­ta is re­leased.

No­var­tis is look­ing to con­sid­er­ably en­hance its mar­ket for this drug, giv­en an ac­cel­er­at­ed ap­proval two years ago as a  sec­ond-line ther­a­py.

Late last year the FDA hand­ed out an ac­cel­er­at­ed first ap­proval to use Ale­cen­sa for pa­tients who had seen their dis­ease progress while on Xalko­ri.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

Mod­er­na says Covid-19 vac­cine boost­er in­creased im­mune re­sponse against vari­ants of con­cern

About a month after announcing their variant-specific Covid-19 vaccine boosters showed promising results in mice, Moderna says it now has some human data to back it up.

Volunteers given a booster shot about six to eight months after receiving their second dose saw increased antibody levels against SARS-CoV-2 and two variants of concern: B.1.351, which was first identified in South Africa, and P.1, first identified in Brazil, the company said on Wednesday.

FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.

Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.