In a come­back, Por­to­la scores FDA ac­cel­er­at­ed ap­proval for once-re­ject­ed An­dexxa

Some­how stay­ing on the good side of the FDA, Por­to­la $PT­LA has scored an elu­sive reg­u­la­to­ry win for their once-re­ject­ed drug an­dex­anet al­fa.

The an­ti-an­ti­co­ag­u­lant — to be mar­ket­ed as An­dexxa — won the OK via the ac­cel­er­at­ed ap­proval path­way, mean­ing that Por­to­la will have to run a post-mar­ket­ing tri­al to demon­strate an abil­i­ty to con­trol bleed­ing in pa­tients.  The ap­proval was based on da­ta from two Phase III stud­ies, con­duct­ed among healthy vol­un­teers, that showed An­dexxa could re­verse an­ti­co­ag­u­lant ac­tiv­i­ty of the Fac­tor Xa in­hibitors Xarel­to (ri­varox­a­ban) and Eliquis (apix­a­ban). The drug al­so comes with a black box warn­ing and is in­di­cat­ed for use when re­ver­sal of an­ti­co­ag­u­la­tion is need­ed due to life-threat­en­ing or un­con­trolled bleed­ing.

Bill Lis

Por­to­la has fought long and hard for this win. It first sub­mit­ted an NDA for An­dexxa late 2015, on­ly to be caught off guard with an FDA re­jec­tion. At the time, the South San Fran­cis­co biotech said the agency was look­ing for more in­for­ma­tion on man­u­fac­tur­ing and how the an­ti­dote works in oth­er blood thin­ning drugs, such as edox­a­ban (Savaysa) and enoxa­parin (Lovenox).

The team filed again in Au­gust 2017, ex­act­ly a year af­ter it got the com­plete re­sponse let­ter. Reg­u­la­tors have since pushed back the ac­tion date from Feb­ru­ary to May 4, and Por­to­la hint­ed in a re­cent earn­ings call that there could be fur­ther de­lays as the FDA re­quest­ed fur­ther da­ta, send­ing stock down. But noth­ing came out of that warn­ing, and Por­to­la spread word about the OK late Thurs­day — right be­fore the orig­i­nal ac­tion date.

A spokesper­son told me that the CRL Por­to­la re­ceived in 2016 “fo­cused al­most sole­ly on man­u­fac­tur­ing con­cerns, which Por­to­la ad­dressed.”

“An­dexxa is a break­through prod­uct, and as such, clin­i­cal was much faster than man­u­fac­tur­ing,” they wrote. “In oth­er words, at that time, man­u­fac­tur­ing had to catch up with clin­i­cal.”

In a lengthy press re­lease, Por­to­la did not elab­o­rate on how it re­solved the FDA’s pre­vi­ous con­cerns, choos­ing in­stead to high­light the mar­ket po­ten­tial of its re­ver­sal agent in an era when Fac­tor Xa in­hibitors have be­come in­creas­ing pop­u­lar. One of them is Por­to­la’s own Bevyxxa, whose ap­proval raised some eye­brows since it bare­ly failed a piv­otal tri­al. Eu­ro­pean reg­u­la­tors gave it a thumbs down.

“We are proud that An­dexxa is a first-in-class med­i­cine dis­cov­ered in our labs,” said CEO Bill Lis. “We re­main com­mit­ted to our sci­en­tif­ic lead­er­ship in the fields of throm­bo­sis and hema­to­log­ic can­cers.”

The com­pa­ny now plans to launch an ear­ly sup­ply pro­gram of “Gen­er­a­tion 1” An­dexxa in June, with broad­er com­mer­cial launch an­tic­i­pat­ed in ear­ly 2019, as­sum­ing the FDA green­lights the man­u­fac­tur­ing process of its Gen­er­a­tion 2 prod­uct.

Mean­while, the post-mar­ket­ing tri­al is slat­ed to be­gin in 2019 and be re­port­ed in 2023.

Por­to­la shares edged up 3% in pre-mar­ket trad­ing. Ac­cord­ing to Eval­u­atePhar­maAn­dexxa car­ries a 2022 sell­side rev­enue fore­cast of $749m.

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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Eisai and METAvivor plan to debut the latest 'This is MBC' campaign at the San Antonio Breast Cancer Symposium (SABCS).

Ei­sai re-ups metasta­t­ic breast can­cer aware­ness cam­paign with strik­ing pa­tient pho­tographs

Eisai is debuting the newest ads in its long-running “This is MBC” campaign this week. In what’s become an annual tradition, Eisai and metastatic breast cancer advocacy partner METAvivor will show the striking photographs of people living with metastatic breast cancer first at the San Antonio Breast Cancer Symposium (SABCS).

The new “Imagine” campaign features 12 patients photographed around waterfalls to symbolize that same kind of sudden drop into a pool that MBC causes in a person’s life, said Beth Fairchild, co-founder of #CancerCulture who was the president of METAvivor six years ago when the campaign began. Fairchild, who is living with MBC, has helped create all of the annual “This is MBC” campaigns.

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Pfiz­er and BioN­Tech look to toss Mod­er­na patent suit, call­ing claims 'unen­force­able'

Pfizer and BioNTech took a swing at Moderna’s Covid-19 patent claims in Massachusetts federal court on Monday, calling them “invalid,” “overbroad” and “unenforceable.”

The defendants also filed counterclaims against the Cambridge, MA-based biotech, seeking a dismissal of the case, recovery of court fees and an official judgment invalidating Moderna’s claims.

Moderna sued Pfizer and BioNTech back in August, alleging that the partners’ Covid-19 vaccine Comirnaty copied parts of Moderna’s vaccine technology patented before the pandemic, when it was developing an mRNA vaccine for MERS, another respiratory illness.

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Glen­mark hit with warn­ing let­ter over pro­ce­dures, qual­i­ty con­trol is­sues at In­dia man­u­fac­tur­ing plant

The generics producer Glenmark Pharmaceuticals has been handed a warning letter by US regulators.

The letter, which was sent to the manufacturer on Nov. 22, noted issues from an inspection over the summer at Glenmark’s facility in the town of Colvale, India, in the state of Goa.

According to the letter, the FDA found that Glenmark’s investigation of rejected batches of drugs “failed to extend to other batches, dosage strengths, and drug products.” The warning letter also noted that the site had failed to establish “adequate written procedures” for production and process control to ensure drugs have the correct strength, quality and purity.

Klick Health is lighting the way, literally, this holiday season to encourage connection for lonely seniors in long-term care facilities.

Klick Health an­nu­al hol­i­day spot­light se­nior lone­li­ness and the pow­er of con­nec­tion

Every year Klick Health leans into a cause for the holidays, and this year it’s highlighting the sometimes lonely season for seniors. So Klicksters, as employees call themselves, decided to brighten one nursing home community in hopes of inspiring others to do the same.

Klick literally lit up the Tony Stacey Centre for Veterans Care, a long-term care home in Toronto where 75% of residents receive no visitors during the holiday season. The agency brought staff and family along with lighting crews and musicians for a “Light the Way” event, creating a video of the experience debuting on Tuesday.

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Sum­i­to­vant sub­sidiaries En­zy­vant and Al­ta­vant merge in­to com­bined com­pa­ny

Two Sumitovant Biopharma entities are merging under one name, effective immediately.

Enzyvant Therapeutics and Altavant Sciences announced they have merged to form a singular entity focused on developing therapies for patients with rare diseases. The combined company will keep the name Enzyvant and along with clinical development will eventually include in-house manufacturing.

Bill Symonds, the current CEO of both Altavant and Enzyvant, is now CEO of the merged company.

Eu­ro­pean Com­mis­sion lays ground­work to un­wind Il­lu­mi­na's $7B+ Grail merg­er

The European Commission has recommended steps that — though not yet final — would require Illumina to “swiftly” unwind its controversial $7.1 billion Grail buyout.

The Commission delivered a “statement of objections” on Monday, detailing the process Illumina would need to take in divesting Grail, its blood testing spinout launched in 2016. Illumina re-acquired Grail back in August, despite criticism from both the FTC and EU.

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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