FDA stamps fast OK on Astel­las' pi­o­neer­ing FLT3 AML drug gilter­i­tinib, ex­pand­ing on a record year for new ap­provals

Astel­las has picked up boast­ing rights to gain­ing the first FDA ap­proval of a FLT3 in­hibitor for a spe­cif­ic group of FLT3 pos­i­tive acute myeloid leukemia pa­tients — which ac­counts for close to a third of the pop­u­la­tion.

Back in May the Japan­ese phar­ma re­port­ed that the agency had of­fered a snap 6-month re­view on its fast-track pro­gram for gilter­i­tinib, some­thing that’s be­come quite com­mon in the on­col­o­gy group.

That’s good news for the 30% or so of the 19,000 new­ly di­ag­nosed AML cas­es each year that will be linked to the ge­net­ic mu­ta­tion. The drug has been in a piv­otal study where in­ves­ti­ga­tors have been study­ing it’s abil­i­ty to cut re­laps­es in the pa­tient pop­u­la­tion.

The drug will be sold as Xospa­ta, and Astel­las set the whole­sale price at $22,500 for 30 days, which would trans­late to $270,000 for a year of ther­a­py — though pay­ers ne­go­ti­ate down from that fig­ure. In a state­ment, the com­pa­ny said that their in­ter­im analy­sis of the piv­otal pro­gram tracked a me­di­an du­ra­tion of re­sponse of 4.6 months.

Alexan­der Ed­ward Perl

This OK de­liv­ers a “new, high­ly-ef­fec­tive, and well-tol­er­at­ed treat­ment op­tion to the clin­ic for a group of tru­ly high-risk pa­tients who, un­til to­day, had no spe­cif­ic ther­a­pies avail­able be­yond chemother­a­py to treat their dis­ease,” says Alexan­der Perl, an as­so­ciate pro­fes­sor of Hema­tol­ogy-On­col­o­gy at the Uni­ver­si­ty of Penn­syl­va­nia. Perl has been lead­ing the clin­i­cal tri­al pro­gram for this drug.

“Al­though we’re wait­ing for the fi­nal analy­sis of (the Phase III study) AD­MI­RAL, the avail­able da­ta with gilter­i­tinib show few­er and milder side ef­fects than typ­i­cal­ly is seen with tra­di­tion­al chemother­a­py,” Perl added, not­ing that they al­so tracked a sig­nif­i­cant­ly high­er rate of com­plete re­spons­es.

The ap­proval marks the FDA’s 54th OK of the year as it goes deep­er in­to record ter­ri­to­ry af­ter pass­ing the old record set in 1996.


2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Spe­cial re­port 2022: Meet 20 women blaz­ing trails in bio­phar­ma R&D

When you run a special report for a fourth year, it can start feeling a little bit like a ritual. You go through the motions — in our case opening up nominations for top women in biopharma R&D and reviewing more than 500 entries — you make your choices of inclusion and exclusion. You host a ceremony.

But then things happen that remind you why you do it in the first place. Perhaps a Supreme Court rules to overturn the constitutional right to abortion and a group of women biotech leaders makes it clear they strongly dissent; perhaps new data on gender diversity in the industry come out that look all too similar to the old ones, suggesting women are still dramatically underrepresented at the top; perhaps protests and conflicts around the world put in stark terms the struggles that many women still face in earning the most basic recognition.

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Phar­ma rep­u­ta­tion re­tains 'halo' even as pan­dem­ic me­dia cov­er­age re­cedes — sur­vey

The Covid-19 halo effect on the pharma industry is continuing, according to a new global study from Ipsos. The annual survey for the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) finds considerable goodwill from consumers across measures of trust, cooperation with governments, and advancing research and drug development.

“Despite the pandemic in many countries no longer being the top of mind concern generally – although it does remain the top concern as a health issue – the industry’s reputation has remained positive,” said Ipsos research director Thomas Fife-Schaw.

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Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Prometheus moves to raise cash hours af­ter PhII da­ta leads to stock surge

After releasing better-than-anticipated data on two mid-stage studies Wednesday morning, Prometheus Biosciences’ CEO said the company would “take some time to assess” its next financing options.

It only needed about seven hours. Wednesday afternoon after the market closed, the biotech announced it would seek $250 million through an equity offering as the company looks to edge out anti-TL1A competitor Pfizer and its new partner Roivant.

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Enhertu researcher Ian Krop speaks during Wednesday's SABCS press conference (MedMeetingImages/Todd Buchanan via SABCS)

SABCS roundup: No­var­tis shows two-year PFS in breast can­cer sub­groups; As­traZeneca re­veals more En­her­tu da­ta

The San Antonio Breast Cancer Symposium is taking place this week, and so far, some of the Big Pharmas are turning out new trial data about some of the biggest drugs in the space.

First off, Novartis announced that its drug, Kisqali, showed about a year of progression-free survival in patients with different types of first-line metastatic breast cancer. The CDK 4/6 drug was first approved by the FDA in 2017, setting it up in direct competition against Pfizer’s Ibrance.

Big Phar­mas team up with lo­cal Sin­ga­pore or­ga­ni­za­tions to boost man­u­fac­tur­ing

Singapore has long established itself as a major hub for pharma manufacturing, and now several big players are looking to further cement their presence in the Lion City.

Takeda, Sanofi and GSK are forming a partnership with the Singapore-based Agency for Science, Technology and Research (A*STAR) and several local academic institutions, including the National University of Singapore, Nanyang Technological University, Singapore, its enterprise company called NTUitive and Singapore Institute of Technology to provide a greater boost to the manufacturing of biologics.

Piper Trelstad, head of CMC, Bill & Melinda Gates Medical Research Institute

Q&A with Gates leader: Women tak­ing on more roles in phar­ma man­u­fac­tur­ing, but still work to do

More and more women are driving innovation and taking leadership roles in biotech – as evidenced today in the release of Endpoints News’ list of the top 20 women in the R&D world – but those gains are beginning to extend across pharma sectors.

In pharma manufacturing in the US today, around 46% of all roles are occupied by women, according to the US Bureau of Labor Statistics for 2021. And according to a Bloomberg report, women’s roles across manufacturing roles had a massive boost after the start of the pandemic.