In a set­back, Glob­al Blood Ther­a­peu­tics scraps IPF pro­gram as lead drug floun­ders in proof-of-con­cept tri­als

Ted Love

It’s all or noth­ing for Glob­al Blood Ther­a­peu­tics’ clin­i­cal tri­al pro­gram for sick­le cell dis­ease.

The South San Fran­cis­co-based biotech an­nounced this morn­ing that it is scrap­ping an am­bi­tious ear­ly-stage ef­fort to de­vel­op their lead drug GBT440 as a ther­a­py for hy­pox­emia — low lev­els of oxy­gen in the blood of pa­tients suf­fer­ing from id­io­path­ic pul­monary fi­bro­sis. That leaves sick­le cell dis­ease as the sole in­di­ca­tion now be­ing stud­ied in the clin­ic.

Glob­al Blood’s stock $GBT swift­ly took a 12% hit Mon­day morn­ing in pre-mar­ket trad­ing as in­vestors sized up the set­back.

Ac­cord­ing to re­searchers, they were able to track an in­crease in the lev­el of oxy­gen in the blood of peo­ple tak­ing the ther­a­py, re­view­ing two Phase IIa stud­ies and a Phase I in healthy vol­un­teers. But that im­prove­ment in oxy­gen lev­els didn’t ac­tu­al­ly trans­late in­to clin­i­cal­ly mean­ing­ful ben­e­fits for the pa­tients in the proof-of-con­cept study.

Their Phase III study for sick­le cell dis­ease is ex­pect­ed to read out in 2019.

Last March Reuters re­port­ed that the Dan­ish phar­ma com­pa­ny No­vo Nordisk was kick­ing the tires at GBT, pos­si­bly in­ter­est­ed in mak­ing a play for the biotech. But noth­ing came out of that, yet. That sto­ry hit 5 months af­ter No­var­tis bought out sick­le cell ri­val Se­lexys, which had a mid-stage drug in the clin­ic for symp­toms of sick­le cell dis­ease. GBT440, though, looks to treat a root cause of the dis­ease.

“From the out­set, we set a high bar for suc­cess in our IPF pro­gram. The re­sults re-af­firm our con­fi­dence in the mech­a­nism of ac­tion of GBT440. How­ev­er, the da­ta from these proof-of-con­cept stud­ies did not demon­strate suf­fi­cient over­all clin­i­cal ben­e­fit to jus­ti­fy con­tin­u­ing the pro­gram,” said GBT CEO Ted Love.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

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Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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